Restoration of muscle strength in dystrophic muscle by angiotensin-1-7 through inhibition of TGF-β signalling

Hum Mol Genet. 2014 Mar 1;23(5):1237-49. doi: 10.1093/hmg/ddt514. Epub 2013 Oct 24.

Abstract

Duchenne muscular dystrophy (DMD) is the most common inherited neuromuscular disease, and is characterized by the lack of dystrophin, muscle wasting, increased transforming growth factor (TGF)-β Smad-dependent signalling and fibrosis. Acting via the Mas receptor, angiotensin-1-7 [Ang-(1-7)], is part of the renin-angiotensin system, with the opposite effect to that of angiotensin II. We hypothesized that the Ang-(1-7)/Mas receptor axis might protect chronically damaged tissues as in skeletal muscle of the DMD mouse model mdx. Infusion or oral administration of Ang-(1-7) in mdx mice normalized skeletal muscle architecture, decreased local fibrosis and improved muscle function in vitro and in vivo. These positive effects were mediated by the inhibition of TGF-β Smad signalling, which in turn led to reduction of the pro-fibrotic microRNA miR-21 concomitant with a reduction in the number of TCF4 expressing fibroblasts. Mdx mice infused with Mas antagonist (A-779) and mdx deficient for the Mas receptor showed highly deteriorated muscular architecture, increased fibrosis and TGF-β signalling with diminished muscle strength. These results suggest that this novel compound Ang-(1-7) might be used to improve quality of life and delay death in individuals with DMD and this drug should be investigated in further pre-clinical trials.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Angiotensin I / administration & dosage
  • Angiotensin I / pharmacology*
  • Animals
  • Disease Models, Animal
  • Extracellular Matrix / metabolism
  • Fibroblasts / drug effects
  • Fibroblasts / metabolism
  • Fibrosis
  • Humans
  • Male
  • Mice
  • Mice, Inbred mdx
  • Mice, Knockout
  • MicroRNAs / genetics
  • MicroRNAs / metabolism
  • Muscle Strength / drug effects*
  • Muscle, Skeletal / drug effects
  • Muscle, Skeletal / metabolism
  • Muscle, Skeletal / pathology
  • Muscular Dystrophy, Duchenne / genetics
  • Muscular Dystrophy, Duchenne / metabolism*
  • Peptide Fragments / administration & dosage
  • Peptide Fragments / pharmacology*
  • Receptors, Cell Surface / antagonists & inhibitors
  • Signal Transduction / drug effects*
  • Transforming Growth Factor beta / metabolism*

Substances

  • MIRN21 microRNA, mouse
  • MicroRNAs
  • Peptide Fragments
  • Receptors, Cell Surface
  • Transforming Growth Factor beta
  • angiogenin receptor
  • Angiotensin I
  • angiotensin I (1-7)